Summary

Eligibility
for males ages 10 years and up (full criteria)
Location
at UC Davis
Dates
study started
estimated completion
Principal Investigator
by Craig McDonald, MD (ucdavis)

Description

Summary

HOPE-3 is a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

Official Title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy

Details

Upto 68 eligible study participants will be randomized to either CAP-1002 or placebo in a 1:1 ratio. - The trial will include visits at Screening, Baseline/Day 1, Week 4, and Months 3, 6, 9, and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3, 6, and 9. - Safety evaluations will include adverse events, concomitant medications, physical exam, vital signs, 12-lead ECG, and clinical laboratory testing. - Efficacy will be evaluated in the Performance of the Upper Limb, Egen Klassifikation Scale, North Star Ambulatory Assessment (ambulatory subjects only), 10 meter walk test (ambulatory subjects only), cardiac MRI, blood collection and quality of life.

Keywords

Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Neuromuscular Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Nervous System Diseases Duchenne Muscular Dystrophy Cell Therapy Performance of the Upper Limb Ambulatory Non-Ambulatory CAP-1002

Eligibility

You can join if…

Open to males ages 10 years and up

  1. Male subjects at least 10 years of age at time of consent who are willing and able to provide informed consent to participate in the trial and diagnosed with DMD as confirmed by the Investigator
  2. Genetically confirmed DMD
  3. Reduced ability to walk/run (if ambulatory)
  4. Treatment with systemic glucocorticoids for at least 12 months and at a stable dose at least 6 months prior to study participation, except for weight-based or toxicity-related adjustments
  5. Current and up-to-date immunizations

You CAN'T join if...

  1. Left ventricular ejection fraction (LVEF) < 35%
  2. Elbow-flexion contractures > 30° in both extremities
  3. Body mass index (BMI) > 45
  4. Percent predicted forced vital capacity (FVC%) < 35%

Location

  • University of California, Davis
    Sacramento California 95817 United States

Lead Scientist at University of California Health

  • Craig McDonald, MD (ucdavis)
    Professor, Physical Medicine and Rehabilitation. Authored (or co-authored) 205 research publications.

Details

Status
not yet accepting patients
Start Date
Completion Date
(estimated)
Sponsor
Capricor Inc.
ID
NCT05126758
Phase
Phase 3
Study Type
Interventional
Last Updated